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Carcinoma, Squamous Cell of Head and NeckPhase 1NCT02113878

Phase Ib Study of BKM120 With Cisplatin and XRT in High Risk Locally Advanced Squamous Cell Cancer of Head and Neck

A Phase 1 study testing BKM120, Cisplatin and Intensity-modulated radiotherapy (IMRT) in people with Carcinoma, Squamous Cell of Head and Neck and HPV Positive Oropharyngeal Squamous Cell Carcinoma, run by Dana-Farber Cancer Institute at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This research study is evaluating a drug called buparlisib (BKM120) as a possible treatment for locally advanced head and neck squamous cell cancer. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
BKM120, Cisplatin and Intensity-modulated radiotherapy (IMRT)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
23 participants
Where
1 siteUnited States
Study dates
29 Sep 2014to 1 Jan 2022
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Stage III/IV, locally advanced, biopsy proven squamous cell cancer of the head and neck that undergo chemoradiation as their primary treatment with curative intent.
  • Oropharynx (HPV positive and HPV negative), hypopharynx, larynx primaries, nasopharynx as well as those with documented SCC of the cervical lymph nodes, with unknown primaries.
  • >10 pack years of tobacco use
  • Age ≥ 18 years
  • ECOG performance status ≤ 2
  • At least one site of measurable disease
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  • Adequate bone marrow function as shown by: ANC > 1.5 x 109/L, Platelets >100 x 109/L, Hb >9 g/dL
  • Total calcium (corrected for serum albumin) within normal limits
  • Magnesium ≥ the lower limit of normal
  • Potassium within normal limits for the institution.
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) within normal range
  • Serum bilirubin within normal range (or ≤ 1.5 x ULN if liver metastases are present; or total bilirubin ≤ 3.0 x ULN with direct bilirubin within normal range in patients with well documented Gilbert Syndrome)
  • Serum creatinine ≤ 1.5 x ULN or 24-hour clearance ≥ 50 mL/min
  • Serum amylase ≤ ULN
  • Serum lipase ≤ ULN
  • Fasting plasma glucose ≤ 120 mg/dL (6.7 mmol/L)
  • Signed informed consent
  • INR ≤ 2

Who cannot join

  • Distant metastatic disease
  • Less than or equal to 10 pack years of tobacco history
  • Received prior chemotherapy
  • Received prior radiation to the head and neck or adjacent anatomical site
  • Received prior treatment with a P13K inhibitor.
  • Known hypersensitivity to BKM120 or to its excipients
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  • Acute or chronic liver, renal disease or pancreatitis
  • Mood disorders ≥ CTCAE grade 3
  • Diarrhea ≥ CTCAE grade 2
  • Active cardiac disease
  • History of cardiac dysfunction including any of the following:
  • Patient has poorly
  • Impairment of gastrointestinal (GI) function
  • Currently receiving treatment with medication with a known risk to prolong the QT interval or inducing Torsades de Pointes and the treatment cannot either be discontinued or switched to a different medication prior to starting study drug.
  • Chronic treatment with steroids or another immunosuppressive agent.
  • Herbal medications and certain fruits within 7 days prior to starting study drug.
  • Currently treated with drugs known to be moderate and strong inhibitors or inducers of isoenzyme CYP3A, and the treatment cannot be discontinued or switched to a different medication prior to starting study drug. Please refer to Appendix B for a list of prohibited inhibitors and inducers of CYP3A (Please note that co-treatment with weak inhibitors of CYP3A is allowed).
  • Undergone major surgery ≤ 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.
  • Currently taking therapeutic doses of warfarin sodium or any other coumadin-derivative anticoagulant.
  • Women who are pregnant or breast feeding or adults of reproductive potential not employing an effective method of birth control.
  • Known diagnosis of human immunodeficiency virus (HIV) infection
  • History of another malignancy within 3 years, except cured basal cell carcinoma of the skin or excised carcinoma in situ of the cervix

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02115

Other trials in United States

About this study

Official title: A Phase Ib Study of BKM120 With Weekly Cisplatin and Radiotherapy in High Risk Locally Advanced Squamous Cell Cancer of the Head and Neck

This research study is evaluating a drug called buparlisib (BKM120) as a possible treatment for locally advanced head and neck squamous cell cancer.

Read the detailed description
  • This phase Ib study is combining standard chemoradiotherapy with weekly cisplatin and BKM120 to assess tolerability of this combination in high risk patients with locally advanced Squamous Cell Carcinoma of the Head and Neck (SCCHN). The investigators will also obtain preliminary information about the efficacy of this treatment.
  • The participant will receive the study drug buparlisib once daily, by mouth, for 45 days. The participant will be given a study drug-dosing diary for each cycle. It will include special instructions for taking the study drug at home.
  • The investigators are looking for the highest dose of the study drug that can be administered safely without severe or unmanageable side effects, not everyone who participates in this research study will receive the same dose of the study drug. The dose given will depend on the number of participants who have been enrolled in the study before and how well they have tolerated their doses.
  • All participants will receive weekly cisplatin injection. Cisplatin will be given intra-venously (IV) on days: (1, 8, 15, 22, 29, 36 and 43) at DFCI.
  • All participants will receive daily radiotherapy with intensity-modulated radiotherapy (IMRT) for 7 weeks, delivered at DFCI. IMRT is a type of 3-dimensional radiation therapy that uses computer-generated images to show the size and shape of the tumor. Thin beams of radiation of different intensities are aimed at the tumor from many angles. This type of radiation therapy reduces the damage to healthy tissue near the tumor.
  • The investigators would like to keep track of the participant's medical condition. Follow-up will continue every 4 to 12 weeks after the end of treatment for the first year and at the investigator's discretion thereafter.
Being tested
BKM120, Cisplatin, Intensity-modulated radiotherapy (IMRT)
Keywords
Carcinoma, Squamous Cell of Head and Neck, Human Papillomavirus Positive Oropharyngeal Carcinoma, Hypopharyngeal Cancer, Early Invasive Cervical Squamous Cell Carcinoma, Carcinoma of Larynx, Cancer of Nasopharynx
Medical subject headings
Squamous Cell Carcinoma of Head and Neck, Hypopharyngeal Neoplasms, Laryngeal Neoplasms, Nasopharyngeal Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BKM120Drug

    Also called Buparlisib

    BKM120 is a potent and highly specific oral pan-class I PI3K inhibitor.

  • CisplatinDrug

    Also called Platinol-AQ, Platinol

    Cisplatin is a chemotherapy drug

  • Intensity-modulated radiotherapy (IMRT)Radiation

    Also called IMRT

    IMRT is the medical use of ionizing radiation, generally as part of cancer treatment to control or kill malignant cells

Study groups in NCT02113878
Study groupRoleWhat they receive
Dose Level 1Experimental* 40 mg BKM120 will be administered orally daily for 45 days. Starting dose 40 mg. * Cisplatin: Starting Dose 30 mg/m2, given IV, weekly on days: (1, 8, 15, 22, 29, 36 and 43). * Radiotherapy: All participants will receive daily radiotherapy with intensity-modulated radiotherapy (IMRT) for 7 weeks.
Receives: Drug: BKM120; Drug: Cisplatin; Radiation: Intensity-modulated radiotherapy (IMRT)
Dose Level 2Experimental* 40 mg BKM120 will be administered orally daily for 45 days. * Cisplatin: Starting Dose 35 mg/m2, given IV, weekly on days: (1, 8, 15, 22, 29, 36 and 43). * Radiotherapy: All participants will receive daily radiotherapy with intensity-modulated radiotherapy (IMRT) for 7 weeks.
Receives: Drug: BKM120; Drug: Cisplatin; Radiation: Intensity-modulated radiotherapy (IMRT)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum Tolerated Dose of Cisplatin

    The trial uses 3+3 design to determine maximum tolerated dose (MTD).

    Measured: While on treatment, up to 66 days

  2. Maximum Tolerated Dose of BKM120

    The trial uses 3+3 design to determine maximum tolerated dose (MTD).

    Measured: While on treatment, up to 66 days

Secondary outcomes (6)

  1. Best Overall Response

    The best overall response for each patient is given per Response Evaluation Criteria in Solid Tumors Criteria (RECIST 1.1) criteria:

    Complete Response (CR) - Disappearance of all target and non-target lesions and lymph node short axis is <10 mm and no new lesions

    Partial Response (PR)

    • Disappearance of target lesions and >=1 persisting non-target lesion or tumor marker levels above normal limits and no new lesions or
    • >=30% decrease in sum of target lesion diameter and no new lesions

    Stable Disease (SD)

    • Less than 5 mm increase in target lesion diameter unless it results in a >=20% increase in diameter and >=1 persisting non-target lesion or tumor marker levels above normal limits and no new lesions

    Progressive Disease (PD)

    • >20% increase in target lesion diameter, must be an absolute increase of 5mm or
    • Substantial worsening in non-target lesion or
    • New lesion present

    Measured: Measured at end of treatment, up to 66 days

  2. Median Time to Progression

    The median amount of time from baseline to progression and/or death. Patients known to be alive at last contact are censored at last contact. All patients alive at 24 months from baseline are censored at 24 months.

    Measured: Up to 24 months (Progression is defined using RECIST v1.0 as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions)

  3. 24 Month Overall Survival

    The probability of survival at 24 months using Kaplan-Meier estimates. Patients alive at 2 years are censored at 2 years.

    Measured: Up to 24 months

  4. Median Anxiety Score Change

    The median change in score on the Generalized Anxiety Disorder - 7 (GAD-7) questionnaire from baseline to cycle 9.GAD-7 has 7 questions and each question has score from 0 to 3. Sum score of all questions will be used as the final score. The final score ranges from 0 to 21. Higher score represents worse anxiety.

    Measured: From baseline to cycle 9, up to 66 days.

  5. Median Depression Score Change

    The median change in score on the Patient Health Questionnaire - 9 (PHQ-8) from baseline to cycle 9. PHQ-9 has 9 questions and each question has score from 0 to 3. Sum score of all questions will be used as the final score. The final score ranges from 0 to 27. Higher score represents worse depression.

    Measured: From baseline to cycle 9, up to 66 days.

  6. P13K Status by Response

    Phosphoinositide 3-kinase (PI3K) biomarker status, either positive or negative, analyzed using established methods from tumor biopsies. P13K status is reported by response, either responder (CR or PR) or non-responder (SD or PR).

    Measured: Biopsies drawn up to 10 days from registration. Response measured at from baseline to cycle 9, up to 66 days.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Apr 2014 (estimated)
  2. Study start29 Sep 2014
  3. Primary completion6 Dec 2019
  4. Study completion1 Jan 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02113878
Sponsor's protocol ID
14-008
Status last verified
Nov 2024
First submitted
1 Apr 2014
Last updated
5 Nov 2026
Data monitoring committee
Yes
Individual data sharing
No: There are no plans to share the IPD. Cumulative data will be published however.

Publications

  • Glorieux M, Dok R, Nuyts S. The influence of PI3K inhibition on the radiotherapy response of head and neck cancer cells. Sci Rep. 2020 Oct 1;10(1):16208. doi: 10.1038/s41598-020-73249-z.

    PubMed 33004905

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BKM120, Cisplatin and Intensity-modulated radiotherapy (IMRT) in people with Carcinoma, Squamous Cell of Head and Neck and HPV Positive Oropharyngeal Squamous Cell Carcinoma, run by Dana-Farber Cancer Institute at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 5 Nov 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Tolerated Dose of Cisplatin (time frame: While on treatment, up to 66 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02113878, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.

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